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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04457336




Registration number
NCT04457336
Ethics application status
Date submitted
25/06/2020
Date registered
7/07/2020
Date last updated
10/06/2024

Titles & IDs
Public title
A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects With Classic Congenital Adrenal Hyperplasia
Secondary ID [1] 0 0
CAHmelia 203
Secondary ID [2] 0 0
SPR001-203
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Congenital Adrenal Hyperplasia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Tildacerfont/Placebo

Experimental: Tildacerfont Group 1 - Tildacerfont administered daily via oral tablet for 12 weeks at dose level 1

Experimental: Tildacerfont Group 2 - Tildacerfont administered daily via oral tablet for 12 weeks at dose level 2

Experimental: Tildacerfont Group 3 - Tildacerfont administered daily via oral tablet for 70 weeks at dose level 3

Placebo Comparator: Placebo - Placebo administered daily via oral tablet for 12 weeks.


Treatment: Drugs: Tildacerfont/Placebo
Tablet, administered daily
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in androstenedione
Timepoint [1] 0 0
12 weeks
Secondary outcome [1] 0 0
Proportion of subjects who achieve reduction A4 levels
Timepoint [1] 0 0
12 weeks
Secondary outcome [2] 0 0
Proportion of subjects who achieve reduction in 17-OHP
Timepoint [2] 0 0
12 weeks
Secondary outcome [3] 0 0
Effectiveness in reducing TART(s) in Male CAH subjects
Timepoint [3] 0 0
12 weeks

Eligibility
Key inclusion criteria
- Male and female subjects over 18 years old, inclusive

- Has a known childhood diagnosis of classic CAH due to 21-hydroxylase deficiency based
on genetic mutation in CYP21A2 and/or documented elevated 17-OHP and currently treated
with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combination of
the aforementioned GCs)

- Has been on a stable supraphysiologic dose of GC replacement =15 mg/day and =60 mg/day
in HC equivalents

- For subjects with the salt-wasting form of CAH, subject has been on a stable dose of
mineralocorticoid replacement for =1 month before screening
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Has a known or suspected diagnosis of any other known form of classic CAH (not due to
21 hydroxylase deficiency)

- Has a history that includes bilateral adrenalectomy or hypopituitarism

- Has a history of allergy or hypersensitivity to Tildacerfont, any of its excipients,
or any other CRF1 receptor antagonist

- Current treatment with dexamethasone as GC therapy for CAH. Prior treatment with
dexamethasone is allowed as long as the transition to an alternative GC regimen (eg,
HC, prednisone, or prednisolone) has resulted in a stable dose of GC replacement for
=1 month before screening.

- Shows clinical signs or symptoms of adrenal insufficiency

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/08/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
23/05/2024
Sample size
Target
Accrual to date
Final
96
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 0 0
Spruce Study Site - Nedlands
Recruitment hospital [2] 0 0
Spruce study site - Brisbane
Recruitment hospital [3] 0 0
Spruce Study Site - Elizabeth Vale
Recruitment hospital [4] 0 0
Spruce Study Site - Melbourne
Recruitment postcode(s) [1] 0 0
6009 - Nedlands
Recruitment postcode(s) [2] 0 0
- Brisbane
Recruitment postcode(s) [3] 0 0
- Elizabeth Vale
Recruitment postcode(s) [4] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Indiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Maryland
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
Nevada
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Oregon
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Rhode Island
Country [15] 0 0
United States of America
State/province [15] 0 0
South Carolina
Country [16] 0 0
United States of America
State/province [16] 0 0
Tennessee
Country [17] 0 0
United States of America
State/province [17] 0 0
Texas
Country [18] 0 0
Brazil
State/province [18] 0 0
Brasília
Country [19] 0 0
Brazil
State/province [19] 0 0
São Paulo
Country [20] 0 0
Canada
State/province [20] 0 0
Newfoundland and Labrador
Country [21] 0 0
Canada
State/province [21] 0 0
Ontario
Country [22] 0 0
Canada
State/province [22] 0 0
Ottawa
Country [23] 0 0
Canada
State/province [23] 0 0
Sherbrooke
Country [24] 0 0
Denmark
State/province [24] 0 0
Aarhus
Country [25] 0 0
Denmark
State/province [25] 0 0
Copenhagen
Country [26] 0 0
Estonia
State/province [26] 0 0
Tallinn
Country [27] 0 0
Estonia
State/province [27] 0 0
Tartu
Country [28] 0 0
Germany
State/province [28] 0 0
Munich
Country [29] 0 0
Ireland
State/province [29] 0 0
Dublin
Country [30] 0 0
Italy
State/province [30] 0 0
Milan
Country [31] 0 0
Italy
State/province [31] 0 0
Napoli
Country [32] 0 0
Italy
State/province [32] 0 0
Rome
Country [33] 0 0
Italy
State/province [33] 0 0
Torino
Country [34] 0 0
Korea, Republic of
State/province [34] 0 0
Seoul
Country [35] 0 0
Latvia
State/province [35] 0 0
Riga
Country [36] 0 0
Lithuania
State/province [36] 0 0
Kaunas
Country [37] 0 0
Netherlands
State/province [37] 0 0
Nijmegen
Country [38] 0 0
Poland
State/province [38] 0 0
Kraków
Country [39] 0 0
Poland
State/province [39] 0 0
Warsaw
Country [40] 0 0
Romania
State/province [40] 0 0
Bucharest
Country [41] 0 0
Spain
State/province [41] 0 0
Barcelona
Country [42] 0 0
Spain
State/province [42] 0 0
Madrid
Country [43] 0 0
Spain
State/province [43] 0 0
Sevilla
Country [44] 0 0
Spain
State/province [44] 0 0
Tarragona
Country [45] 0 0
Sweden
State/province [45] 0 0
Falun
Country [46] 0 0
Sweden
State/province [46] 0 0
Stockholm
Country [47] 0 0
Switzerland
State/province [47] 0 0
Saint Gallen
Country [48] 0 0
Switzerland
State/province [48] 0 0
Zürich
Country [49] 0 0
Turkey
State/province [49] 0 0
Istanbul
Country [50] 0 0
United Kingdom
State/province [50] 0 0
Birmingham

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Spruce Biosciences
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
An investigation of the efficacy and safety of up to 70 weeks of treatment with Tildacerfont
in subjects with classic CAH who have elevated biomarkers at baseline on their current GC
regimen. Optional open label treatment extension period up to 240 weeks with 200mg
Tildacerfont QD.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04457336
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Kyriakie Sarafoglou, M.D
Address 0 0
Dept. of Pediatrics, Divisions of Endocrinology and Genetics & Metabolism, Univ. of Minnesota
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04457336